On March 4, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, reported financial results for the full year 2020 and provided a fourth quarter business update (Press release, argenx, MAR 4, 2021, View Source [SID1234576106]).

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"2020 was an exceptional year for argenx, marked by execution across the organization and highlighted by the positive results of our pivotal Phase 3 ADAPT trial. We have started 2021 on strong footing with the FDA’s acceptance for review of the BLA for efgartigimod, bringing us one step closer to offering a new therapy option to people living with gMG. In preparation for our first commercial launch, we remain committed to a series of educational and engagement efforts with patients, physicians and payors on FcRn as a target and the unmet disease burden that gMG patients face," said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"With proof-of-concept established for efgartigimod in four indications and enrollment on track to begin in the fifth and sixth indications this year, we are building out a broad development plan with our FcRn antagonist. In addition, we expect Phase 1 healthy volunteer data mid-year from our C2 antagonist ARGX-117, our second program in severe autoimmunity, solidifying our commitment to continued pipeline expansion with antibody-based medicines that have potential in multiple autoimmune indications," concluded Mr. Van Hauwermeiren.

FOURTH QUARTER 2020 AND RECENT BUSINESS UPDATE

BLA for efgartigimod accepted for review by FDA; on track with commercial and regulatory preparations in the U.S., Japan, the EU and China.

BLA for IV efgartigimod for treatment of gMG accepted for review by FDA with action date set for December 17, 2021 under Prescription Drug User Fee Act (PDUFA)
Japanese Marketing Authorization Application (J-MAA) expected to be filed with Pharmaceuticals and Medical Devices Agency (PMDA) in first half of 2021 with anticipated Japan commercial launch in 2022
MAA expected to be filed with European Medicines Agency (EMA) in second half of 2021
Zai Lab Limited to discuss potential accelerated regulatory pathway for approval in China with National Medical Products Administration (NMPA)
Commercial readiness activities on track, including:
Build-out of supply chain to ensure ample commercial product available at launch to meet early demand
Hiring of experienced, neurology-focused sales team with aim to have approximately 70 sales representatives in place for launch
Ongoing education efforts with key stakeholder groups, including patients, physicians and payors
Launched pre-approval access program (PAA) in the U.S. and Europe to open availability of efgartigimod to people living with gMG who have a high degree of unmet clinical need and are not able to participate in a clinical trial
Seven global trials to be ongoing in 2021 of efgartigimod across IV and subcutaneous (SC) formulations; proof-of-concept now demonstrated in four indications, which strategically fit within growing commercial franchises.

ADAPT-SC: Enrollment ongoing in registrational trial evaluating non-inferiority based on pharmacodynamic effect of SC efgartigimod compared to IV efgartigimod for treatment of gMG; trial expected to enroll approximately 50 patients
ADHERE: Enrollment ongoing in registrational trial evaluating SC efgartigimod for treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) following interim analysis of safety data as well as efficacy assessments that surpassed pre-defined "GO" threshold; trial expected to enroll approximately 130 patients
ADVANCE and ADVANCE-SC: Registrational trials ongoing of IV and SC efgartigimod for treatment of primary immune thrombocytopenia (ITP); trials expected to each enroll approximately 156 patients
ADDRESS: Registrational trial ongoing of SC efgartigimod for treatment of pemphigus (vulgaris and foliaceus); trial expected to enroll approximately 150 patients
Enrollment in fifth and sixth indications to begin in 2021
Agreement with Zai Lab Limited expected to expand and accelerate global development of efgartigimod, including into additional autoimmune indications

Data expected mid-2021 from Phase 1 healthy volunteer trial of ARGX-117, a potential first-in-class C2 antagonist and second program with broad applicability in severe autoimmunity.
-Trial to evaluate safety and tolerability of single and multiple ascending doses of IV and SC ARGX-117, and to identify dose to take forward into potential Phase 2 proof-of-concept trials, including for multifocal motor neuropathy (MMN)

Combination trials of cusatuzumab remain ongoing for treatment of acute myeloid leukemia (AML) as part of global collaboration and licensing agreement with Cilag GmbH International, an affiliate of Janssen.

Data update from Phase 2 CULMINATE trial evaluating cusatuzumab in combination with azacitidine for treatment of newly diagnosed AML to be presented in peer-reviewed forum
Decision to initiate additional cusatuzumab studies under collaboration will be determined following review of all available data including ongoing Phase 1b ELEVATE trial (NCT04150887), which is evaluating cusatuzumab in combination with venetoclax and azacitidine for treatment of newly diagnosed AML
Immunology Innovation Program (IIP) continues to drive pipeline expansion by identifying potential value-creation opportunities through collaboration with leading disease biologists.

Preclinical work ongoing in early-stage pipeline, including ARGX-118, ARGX-119 and ARGX-120
17 discovery programs under evaluation that emerged from IIP
argenx continues its transition to a global, integrated, immunology organization.

Geneva office opened to support commercial infrastructure ahead of expected EU launch of efgartigimod
Planned transition agreement in place for Chief Financial Officer Eric Castaldi as part of evolution to commercial-stage company; recruitment efforts ongoing for U.S.-based successor
Yvonne Greenstreet, President and Chief Operating Officer of Alnylam, has been nominated to Board of Directors to fill position of Dr. David Lacey, who intends to transition to an advisory role for the Company
Completed public offering of 3,593,750 ordinary shares in February 2021 with gross proceeds of $1.15 billion

DETAILS OF FINANCIAL RESULTS

Cash and cash equivalents and current financial assets totaled €1,627.0 million on December 31, 2020, compared to €1,335.8 million on December 31, 2019. The increase in cash and cash equivalents and current financial assets resulted primarily from the closing of a global offering in May 2020, including a U.S. offering and a European private placement, which resulted in the receipt of €778.1 million in gross proceeds, decreased by €47.4 million of underwriter discounts and commissions, and offering expenses, partially offset by net cash flows used in operating activities.

Revenue decreased by €33.4 million for the year ended December 31, 2020 to €36.4 million, compared to €69.8 million for the year ended December 31, 2019. The decrease was due to the milestone payments following the first-in-human clinical trial with ABBV-151 under the AbbVie collaboration which was achieved in the year ended December 31, 2019, partly offset by revenue recognition of the transaction price related to the Janssen collaboration. The increase in other income is primarily driven by increased research and development incentives and higher payroll tax rebates for employing certain highly qualified research and development personnel.

Research and development expenses increased by €127.8 million for the year ended December 31, 2020 to €325.5 million, compared to €197.7 million for the year ended December 31, 2019. The increase resulted primarily from higher external research and development expenses, primarily related to the efgartigimod program in various indications, cusatuzumab program and other clinical and preclinical programs. Furthermore, the personnel expenses increased due to a planned increase in headcount.

Selling, general and administrative expenses totaled €149.4 million for the year ended December 31, 2020, compared to €64.6 million for the year ended December 31, 2019. The increase primarily resulted from higher personnel expenses and consulting fees related to the preparation of a possible future commercialization of argenx’s lead product candidate efgartigimod.

For the year ended December 31, 2020, financial expenses, which is the net of primarily interest received and changes in fair value of invested funds, amounted to €1.4 million, compared to a financial income of €14.3 million for the year ended December 31, 2020. Financial expenses correspond mainly to the decrease in net asset value of money invested funds following the impact of the COVID-19 outbreak on the financial markets.

Exchange losses totaled €107.0 million for the year ended December 31, 2020, compared to an exchange gain of €6.1 million for the year ended December 31, 2019. The unfavorable change is mainly attributable to unrealized exchange rate losses on cash and cash equivalents and current financial asset position in U.S. dollars.

FINANCIAL GUIDANCE

Based on current plans to fund anticipated operating expenses and capital expenditures, argenx expects its cash burn to increase significantly in 2021, approximately doubling compared to 2020. The increased spend will support the Company’s transition to an integrated immunology company, including the build-out of global commercial infrastructure and drug product inventory ahead of the expected launch of efgartigimod in gMG in the U.S, the advancement of its clinical-stage pipeline, including seven expected global trials of efgartigimod, and the continued investment in its Immunology Innovation Program.

EXPECTED 2021 FINANCIAL CALENDAR

May 14, 2021: Q1 2021 financial results and business update
July 29, 2021: HY 2021 financial results and business update
October 28, 2021: Q3 2021 financial results and business update
CONFERENCE CALL DETAILS
The full year 2020 results and fourth quarter business update will be discussed during a conference call and webcast presentation today at 2:30 pm CEST/8:30 am ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website.

On March 4, 2021 NANOBIOTIX (Euronext : NANO –– NASDAQ: NBTX – the ‘‘Company’’), a clinical-stage biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported that the Company has reached an agreement with PharmaEngine, Inc. ("PharmaEngine") to terminate the License and Collaboration agreement that the Company and PharmaEngine entered into in August 2012 (Press release, Nanobiotix, MAR 4, 2021, View Source [SID1234576122]).

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As previously disclosed in the Nanobiotix prospectus filed with the U.S. Securities and Exchange Commission on December 11, 2020, in November 2020, Nanobiotix notified PharmaEngine of a material breach of the terms of the License and Collaboration agreement. In a letter dated December 1, 2020, PharmaEngine responded to the Company’s notification of material breach, denying a material breach of the License and Collaboration agreement, and asserting certain material breaches of that agreement by Nanobiotix. After discussion between the two parties, this agreement to terminate the License and Collaboration agreement represents a full resolution of outstanding disagreements over a number of issues with respect to the development of NBTXR3 in the Asia-Pacific region.

The License and Collaboration agreement provided PharmaEngine exclusive rights to further the development of NBTXR3 in the Asia-Pacific region. While both Nanobiotix and PharmaEngine believe in the potential of NBTXR3 to improve treatment outcomes for patients with cancer, the parties have had disagreements regarding the optimal strategy for development in the Asia-Pacific region. As such, Nanobiotix and PharmaEngine have mutually agreed to discontinue the collaboration.

Pursuant to their Termination and Release agreement, Nanobiotix will retain all rights to the development and commercialization of NBTXR3 in the Asia-Pacific region. PharmaEngine is to receive payments, not to exceed $5 million in total, upon the completion of various administrative steps in connection with the winding-up of the collaboration.

In the future, PharmaEngine will be entitled to receive a payment of $7.5 million upon a second regulatory approval of NBTXR3 in any jurisdiction of the world for any indication, unless the Company announces a collaboration with a new partner for the Asia-Pacific region within 6 months of the effective date of the agreement. If that occurs, PharmaEngine will be entitled to an immediate $2.5 million payment and will be eligible to receive a payment of the remaining $5 million upon such second regulatory approval of an NBTXR3-containing product. The Company has also agreed to pay royalties to PharmaEngine at low-single digit royalty rates with respect to sales of NBTXR3 in the Asia-Pacific region for a 10-year period commencing on the corresponding first date of sales in the region.

Retention of all rights regarding NBTXR3 will open new near- and long-term possibilities for the Company, and Nanobiotix will evaluate the Asia-Pacific region for potential inclusion in its upcoming global phase III registration trial in head and neck cancer.

About NBTXR3

NBTXR3 is a first-in-class radioenhancer composed of sterile, functionalized, crystalline hafnium oxide nanoparticles. The product candidate is designed to increase the radiotherapy energy deposit inside tumor cells through the nanoparticles’ high atomic number core packaged in the space for interaction with ionizing radiation, and subsequently increase of tumor cell death when compared to radiotherapy alone—without adding toxicity to adjacent healthy tissues. NBTXR3 requires a single, intratumoral administration before the first radiotherapy treatment session, and has the ability to fit into current worldwide standards of radiation care. The primary physical mechanism of action of NBTXR3 activated by radiotherapy could be universal, making it potentially applicable across any solid tumor indication where radiotherapy is a part of standard of care including head and neck, lung, prostate, liver, colorectal, and esophageal cancers. The biological secondary mechanism of action of NBTXR3 activated by radiotherapy has been shown in preclinical studies to prime adaptive immune response, which would potentially bring a new dimension to cancer immunotherapies.

On March 4, 2021 Amgen (NASDAQ: AMGN) and Five Prime Therapeutics (NASDAQ: FPRX), a clinical-stage biotechnology company focused on developing immuno-oncology and targeted cancer therapies, reported an agreement under which Amgen will acquire Five Prime Therapeutics for $38.00 per share in cash, representing an equity value of approximately $1.9 billion (Press release, Amgen, MAR 4, 2021, View Source [SID1234576075]). This acquisition adds Five Prime’s innovative pipeline to Amgen’s leading oncology portfolio.

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Five Prime’s lead asset, bemarituzumab, is a first-in-class, Phase 3 ready anti-FGFR2b antibody with positive data from a randomized, placebo-controlled Phase 2 study in frontline advanced gastric or gastroesophageal junction (GEJ) cancer. Bemarituzumab targets FGFR2b, which has been found to be overexpressed in approximately 30% of patients with non-HER2 positive gastric cancer, as well as other solid tumors.
The bemarituzumab Phase 2 FIGHT trial demonstrated clinically meaningful improvements in progression-free survival (PFS), overall survival (OS) and overall response rate (ORR) in the frontline treatment of patients with advanced gastric or GEJ cancer. Additional analysis showed a positive correlation between efficacy and expression of FGFR2b on tumor cells, confirming both the importance of the FGFR2b target and the activity of bemarituzumab against this target.
This correlation suggests that FGFR2b could play a role in other epithelial cancers, including lung, breast, ovarian and other cancers.
The acquisition of Five Prime also supports Amgen’s international expansion strategy. Gastric cancer is one of the world’s most common forms of cancer and is particularly prevalent in the Asia-Pacific region, where Amgen expects to generate significant volume growth in the coming years. Amgen plans to leverage its presence in Japan and other Asia-Pacific markets to maximize bemarituzumab’s potential. In addition, as part of this transaction, Amgen will receive a royalty percentage on future net sales in Greater China ranging from the high teens to the low twenties from a pre-existing co-development and commercialization agreement between Five Prime and Zai Lab (Shanghai) Co., Ltd.
Five Prime’s additional innovative pipeline programs complement Amgen’s efforts to bring meaningful therapies to oncology patients.
"The acquisition of Five Prime offers a compelling opportunity for Amgen to strengthen our oncology portfolio with a promising late-stage, first-in-class global asset to treat gastric cancer," said Robert A. Bradway, chairman and chief executive officer at Amgen. "We look forward to welcoming the Five Prime team to Amgen and working with them to leverage our best-in-class monoclonal antibody manufacturing capabilities to supply additional clinical materials, as well as expanded production quantities, to realize the full potential of bemarituzumab for even more patients around the world as quickly as possible."

"This is an exciting day for patients who may one day benefit from the promise of bemaritizumab and our full pipeline. I’m so proud of the Five Prime team and the science we’ve pioneered," said Tom Civik, president and chief executive officer of Five Prime. "We see tremendous complementarity between the two companies. Amgen has global reach, world-class resources, and they share our deep passion for science and commitment to patients. I have full confidence that Amgen is the right company to work with us to bring our innovative cancer treatments to patients and to achieve our mission to rewrite cancer."

Transaction Terms
Under the terms of the merger agreement, which was approved by the Boards of Directors of both companies, Amgen will commence a tender offer to acquire all of the outstanding shares of Five Prime’s common stock for $38.00 per share in cash. Following the completion of the tender offer, a wholly-owned subsidiary of Amgen will merge with Five Prime and shares of Five Prime that have not been tendered and purchased in the tender offer will be converted into the right to receive the same price per share in cash as paid in the tender offer (other than shares held by stockholders who properly demand and perfect appraisal rights under Delaware law).

The transaction is expected to close by the end of the second quarter and is subject to customary closing conditions, including the tender of at least a majority of the outstanding shares of Five Prime’s common stock and the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

Amgen reaffirmed its full-year outlook with Revenue guidance of $25.8 to $26.6 billion and non-GAAP EPS guidance of $16.00-$17.00.

Goldman Sachs acted as financial advisor to Amgen and Sullivan & Cromwell LLP as its legal advisor. Lazard acted as financial advisor to Five Prime and Cooley LLP as its legal advisor.

Amgen Webcast Investor Call
Amgen will host a webcast call for the investment community on Thursday, March 4, 2021, at 10:30 a.m. EST. Peter H. Griffith, executive vice president and chief financial officer, David M. Reese, M.D., executive vice president of Research and Development, and Murdo Gordon, executive vice president of Global Commercial Operations at Amgen will participate.

Live audio of the conference call will be broadcast over the internet simultaneously and will be available to members of the news media, investors and the general public. The webcast, as with other selected presentations regarding developments in Amgen’s business given at certain investor and medical conferences, can be accessed on Amgen’s website, www.amgen.com, under Investors. Information regarding presentation times, webcast availability and webcast links are noted on Amgen’s Investor Relations Events Calendar. The webcast will be archived and available for replay for at least 90 days after the event.

About FGFR2b
The fibroblast growth factor (FGF)/fibroblast growth factor receptor (FGFR) pathway is implicated in the development and growth of cancer cells. FGFR2b is a splice variant of FGFR2 which can be found in tumors of epithelial origin. Data from the FIGHT trial suggests that approximately 30 percent of patients with non-HER2 positive gastroesophageal cancers overexpress FGFR2b.1 FGFR2b has also been shown to be overexpressed in numerous other cancers, including lung, breast, ovarian and other cancers.

About Bemarituzumab
Bemarituzumab (anti-FGFR2b) is a first-in-class targeted antibody that blocks fibroblast growth factors (FGFs) from binding and activating FGFR2b, inhibiting several downstream pro-tumor signaling pathways and potentially slowing cancer progression. Five Prime Therapeutics granted an exclusive license to Zai Lab Limited to develop and commercialize bemarituzumab in Greater China, and Zai Lab collaborated with Five Prime Therapeutics on the Phase 2 FIGHT trial in Greater China.

About the FIGHT Trial
The FIGHT study was a randomized, placebo controlled trial that evaluated bemarituzumab plus chemotherapy (mFOLFOX6) versus placebo plus chemotherapy in patients with fibroblast growth factor receptor 2b-positive (FGFR2b+), non HER2 positive frontline advanced gastric or GEJ cancer. The trial enrolled 155 patients in 15 countries across Asia, the European Union, and the United States, with 77 patients randomized to the bemarituzumab arm and 78 patients to the placebo arm.

About Gastric Cancer and GEJ Cancer
Gastric cancer, also known as stomach cancer, is the third most common cause of cancer death worldwide and, excluding non-melanoma skin cancer, the fifth most common cancer worldwide, with over 1,000,000 new cases diagnosed each year.2 For HER2 negative patients, frontline therapy available today is the same systemic chemotherapy available since the 1990s.3,4

On March 4, 2021 Cancer Genetics, Inc. (the "Company") (Nasdaq: CGIX), an emerging leader in novel drug discovery techniques, reported that Jay Roberts, Chief Executive Officer, will present at H.C. Wainwright’s Global Life Sciences Conference (Press release, Cancer Genetics, MAR 4, 2021, View Source [SID1234576091]). The event is being held virtually from March 9-10, 2021.

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Conference Date: March 9-10, 2021 (Tuesday-Wednesday)
On-Demand Starts: 7:00 am ET – Tuesday, March 9, 2021
On-Demand Ends 7:00 pm ET- Wednesday, March 10, 2021
Registration: View Source
Mr. Roberts will highlight the Company’s recent transformational business strategy, including the Company’s proposed merger with StemoniX, Inc., and elaborate on the broader going- forward corporate vision.

If you are an institutional investor and would like to attend the Company’s presentation, please click on the following link (View Source) to register for the H.C. Wainwright Global Life Sciences Conference. Once your registration is confirmed, you will be prompted to log into the conference website and will be able to request a one-on-one meeting with the Company.

Cancer Genetics will also be available for virtual outside 1:1 meetings both during and after the H.C. Wainwright Global Life Sciences Conference. Please contact Jennifer K. Zimmons, Ph.D. [email protected] 917.214.3514 for scheduling.

On March 4, 2021 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported its operational and financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Autolus, MAR 4, 2021, View Source [SID1234576107]).

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"Autolus’ primary focus is on delivering the potential pivotal AUTO1 program and the company starts 2021 in a position of financial strength, having raised a total of $131 million in gross proceeds this quarter, giving us a cash runway into the first half of 2023," said Dr. Christian Itin, chairman and chief executive officer of Autolus. "We are excited about the unique characteristics of AUTO1 and the significant commercial opportunity that adult Acute Lymphoblastic Leukemia represents. Furthermore, we are committed to building additional value by capitalizing on the unique clinical profile of AUTO1 in additional B Cell malignancies and by progressing our pipeline of CAR T cell therapies, including AUTO1/22 in pediatric ALL, AUTO4 in peripheral T cell Lymphoma and AUTO6NG in solid tumors. As such, we expect multiple clinical proof of concept read outs during 2021 and 2022."

Key Pipeline Updates:

AUTO1 in relapsed / refractory (r/r) adult B-Acute Lymphocytic Leukemia (ALL). Positive data from the ALLCAR Phase 1 clinical trial was presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020, demonstrating that, as of the November 12, 2020 data cut-off date, AUTO1 was well tolerated, with no patients experiencing ≥ Grade 3 cytokine release syndrome (CRS). Three patients (15%), all of whom had high leukemia burden (>50% blasts), experienced Grade 3 neurotoxicity (NT) that resolved swiftly with steroids. Of the 19 patients evaluable for efficacy, 16 (84%) patients achieved minimum residual disease (MRD)-negative complete response (CR) at one month. Most notably, the durability of remissions is highly encouraging. Across all treated patients, event free survival (EFS) at six and 12 months is 69% and 52% respectively. Median EFS and overall survival (OS) had not been reached at a median follow up of 16.9 months (range up to 30.5 months). Data from the potential pivotal program, FELIX, is expected in 2022.

AUTO1 in indolent B cell Non-Hodgkin Lymphoma (NHL) (cohort 1), high grade B-NHL (cohort 2) and chronic lymphocytic leukemia (CLL) (cohort 3). Autolus reported positive AUTO1 data at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020. As of the data cut-off date of November 12, 2020, four patients in Cohort 1 had been infused with AUTO1. AUTO1 was well tolerated, with no patients experiencing ≥ Grade 2 CRS and no patients experiencing NT of any grade. All four patients achieved a Complete Metabolic Response (CMR). Autolus is planning to present updated data on AUTO1 in indolent B-cell lymphoma indications at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in June 2021.

AUTO1/22 in pediatric ALL. The first patient was dosed in the extension cohort of the CARPALL clinical trial in Q4 2020. Autolus plans to provide a data update in Q4 2021.

AUTO3 in relapsed/refractory diffuse large B cell lymphoma (DLBCL). Positive data from the Phase 1 ALEXANDER clinical trial was presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020 demonstrating, as of the October 30, 2020 data cut-off date, AUTO3 was well tolerated, with low rates of CRS and NT. Across all 49 patients, there was only one case of Grade 3 CRS with primary infusion, and only three cases of NT were reported, with two being ≥ Grade 3. As of the data cut-off date, none of the patients achieving a complete response (CR) experienced any NT and all cases of NT observed were seen in a setting of disease progression and with confounding factors. Autolus plans to seek a partner for this program.

AUTO4 in Peripheral T Cell Lymphoma (PTCL). AUTO4 will continue, in 2021, to be evaluated in a dose escalation phase of a Phase 1/2 clinical trial in 2021. Autolus expects to provide a next data update in H2 2021.

AUTO5 in Peripheral T Cell Lymphoma. Positive preclinical data were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) II (AACR) (Free AACR Whitepaper) Annual Meeting in June 2020. The data highlight the specificity and selectivity of the Autolus T cell lymphoma product candidate, AUTO5. Autolus expects to initiate a Phase 1 clinical trial in H2 2021.

AUTO6NG in small cell lung cancer (SCLC). Positive preclinical data were presented at the AACR (Free AACR Whitepaper) Annual Meeting in June 2020. Autolus has designed enhancing modules to specifically overcome tumor microenvironment (TME) defenses in solid tumor settings. The new data reported at the AACR (Free AACR Whitepaper) meeting suggest that AUTO6NG can overcome the immune suppressive mechanisms in the TME. Autolus plans to progress AUTO6NG for evaluation in GD2 positive tumors into the clinic in H2 2021.

AUTO7 in prostate cancer. Positive preclinical data were presented at an oral presentation at the AACR (Free AACR Whitepaper) Annual Meeting in June 2020. AUTO7 uses an optimized CAR to target cancer cells expressing PSMA, even at low levels, and includes modules introduced in AUTO6NG, with a module that activates immune responses at the tumor site through limited secretion of IL-12. The data presented at the AACR (Free AACR Whitepaper) meeting demonstrated that AUTO7 is highly potent in cytotoxicity assays against cells expressing PSMA, even at low levels, and demonstrate the feasibility of this multi-modular cell programming approach in overcoming the immunotherapeutic challenges presented by advanced prostate cancer, which is typically otherwise an immunologically cold tumor. Autolus plans to progress AUTO7 into the clinic in H1 2022.

AUTO8 in multiple myeloma. This program will be explored in a first clinical trial starting mid-2021.

Partnerable Coronavirus Disease (COVID-19) Project. Autolus’ research team has developed a potentially universal SARS-CoV2 decoy receptor with virus neutralizing activity against SARS-CoV2 and its variants and also active against SARS-CoV1.
Operational Highlights:

Autolus sold 1,718,506 ADSs under its at-the-market program with Jefferies, for net proceeds of approximately $15.3 million, in January 2021.

Successful closing of a public offering raising net proceeds to Autolus, after underwriting discounts and commissions, of $108.1 million in February 2021, taking total net cash raised in Q1 2021 to approximately $123.4 million.

As announced in Autolus’ business update in January 2021, the company will be prioritizing the AUTO1 program and plans to partner the AUTO3 program before progressing it into the next phase of development.

Also announced in Autolus’ business update in January 2021, the company will adjust its workforce and infrastructure footprint, which will involve an overall reduction in headcount of approximately 20%. The restructuring remains ongoing and Autolus expects to realize cash savings, on an annualized basis, of approximately $15 million per annum once the operational changes are fully implemented.

As previously announced, Dr. Nushmia Khokhar, Senior Vice President, Clinical Development will be leaving the company in mid-March 2021 and Dr. Adam Hacker, Senior Vice President for Regulatory Affairs and Quality, left the Company in January 2021. The company would like to thank Drs. Khokhar and Hacker for their contributions and wishes them well in the future. A search for a Chief Medical Officer is ongoing.

Appointment of Dr Jay T Backstrom to Autolus’ Board of Directors, effective August 1, 2020. Dr Backstrom currently serves as EVP, Head of Research & Development at Acceleron Pharma Inc. and prior to that served as CMO and Head of Regulatory Affairs at Celgene Corporation.
Key Upcoming Clinical Milestones:

AUTO1 updates in 2021 on ALLCAR19 in patients with r/r B-NHL and longer term follow up of the fully enrolled r/r aALL cohort.
AUTO1 – Currently enrolling Phase 1b/2 pivotal study (FELIX) in r/r adult ALL patients with data expected in 2022.
Updates on Phase 1 programs AUTO1/22 in pediatric ALL, as well as AUTO4 in TRBC1+ Peripheral TCL, in 2021.
Phase 1 trials are expected to be initiated in 2021 with AUTO1 in Primary CNS Lymphoma, AUTO5 in TRBC2+ Peripheral TCL, AUTO6NG in Neuroblastoma, and AUTO8 in Multiple Myeloma.
First exploratory allogeneic program expected to enter the clinic in H1 2021.
Financial Results for the Quarter and Year Ended December 31, 2020

Cash at December 31, 2020 totaled $153.3 million, as compared to $210.6 million at December 31, 2019. In January 2021, the company sold 1.7 million ADSs under its Open Market Sales AgreementSM with Jefferies LLC as sales agent, resulting in net proceeds of $15.3 million and in February 2021, the company conducted a public offering of 16,428,572 ADSs representing 16,428,572 ordinary shares, including the exercise in full by the underwriters of their option to purchase an additional 2,142,857 ADSs, at a public offering price of $7.00 per ADS and net proceeds of $108.1 million.

Net total operating expenses for the twelve months ended December 31, 2020 were $168.1 million, net of grant income and license revenue of $1.7 million, as compared to net operating expenses of $146.1 million, net of grant income of $2.9 million, for the same period in 2019.

Research and development expenses increased to $134.9 million for the year ended December 31, 2020 from $105.4 million for the year ended December 31, 2019. Cash costs, which exclude depreciation and amortization as well as share-based compensation, increased to $116.9 million from $83.4 million. The increase in research and development cash costs of $33.5 million consisted primarily of (i) an increase of $8.8 million in compensation and employment related costs, net of lower travel costs, due to an increase in employee headcount to support the advancement of our product candidates in clinical development and lessened travel due to the COVID-19 pandemic, (ii) an increase of $14.4 million in project expenses as a consequence of the advancement of our clinical portfolio which includes research and process development and manufacturing activities necessary to prepare, activate, and monitor clinical trial programs, (iii) an increase of $6.0 million in facilities costs related to the commencement of a lease for an additional manufacturing suite and the continued scaling of manufacturing operations, (iv) an increase of $4.0 million in IT infrastructure and support for information systems related to the conduct of clinical trials and manufacturing operations, (v) an increase of $0.5 million related to legal fees and (vi) an increase of $1.7 million related to cell logistics, which is offset by a reduction in materials purchases of $0.7 million and license fees of $1.1 million.

Non-cash Research & Development costs decreased to $18.1 million for the year ended December 31, 2020 from $22.0 million for the year ended December 31, 2019. The $3.9 million decrease is related to a decrease of $4.8 million share-based compensation expense as a result of a lower fair value of stock options recognized in the period, offset by a $0.9 million increase in depreciation.

General and administrative expenses decreased to $35.0 million for the year ended December 31, 2020 from $39.5 million for the year ended December 31, 2019. Cash costs, which exclude depreciation as well as share-based compensation increased to $27.4 million from $26.6 million. There were increases of $1.3 million related to D&O insurance costs and intellectual property and $0.1 million of facilities cost, offset by decreases of $0.5 million of compensation and other employment related costs and $0.1 million in general office expense.

Non-cash General and Administrative costs decreased to $7.6 million for the year ended December 31, 2020 from $12.9 million for the year ended December 31, 2019. The decrease of $5.3 million is mainly attributed to lower share-based compensation expenses as a result of the lower fair value of share options recognized during the period.

Interest income decreased to $0.5 million for the year ended December 31, 2020 from $2.5 million for the year ended December 31, 2019. This decrease is due to the lower cash balances held during the year combined with lower interest rates for cash held on deposit. Other income decreased to $1.4 million for the year ended December 31, 2020 from $4.5 million for the year ended December 31, 2019 primarily due to a weakening of the U.S. dollar exchange rate relative to the pound sterling. The decrease of $4.6 million in the year ended December 31, 2020 was offset by lease termination gains of $1.5 million.

The Income tax benefit increased to $24.2 million for the year ended December 31, 2020 from $15.2 million for the year ended December 31, 2019 due to additional U.K. research and development tax credits receivable from HMRC. Research and development credits are obtained at a maximum rate of 33.35% of our qualifying research and development expenses, and the increase in the net credit was primarily attributable to an increase in the company’s eligible research and development expenses.

Net loss attributable to ordinary shareholders was $142.1 million for the twelve months ended December 31, 2020, compared to $123.8 million for the same period in 2019. The basic and diluted net loss per ordinary share for the twelve months ended December 31, 2020 totaled $(2.76) compared to a basic and diluted net loss per ordinary share of $(2.88) for the twelve months ended December 31, 2019.

Autolus estimates that its current cash on hand, which includes the recent financings in January and February 2021, will extend the Company’s runway into H1 2023.

Management will host a conference call and webcast at 8:30 am ET/1:30 pm GMT to discuss the company’s financial results and provide a general business update. To listen to the webcast and view the accompanying slide presentation, please go to the events section of Autolus’ website

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 2268057. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 2268057.